DEERFIELD, Ill.--(BUSINESS WIRE)--Baxter International Inc. (NYSE:BAX) today announced pivotal Phase I/III
study results evaluating the safety and efficacy of BAX 326, an
investigational recombinant factor IX (rFIX) protein, for the treatment
and prophylaxis of bleeding episodes for patients with hemophilia B over
12 years of age. The data were presented at the 54th Annual
Meeting and Exposition of the American Society of Hematology (ASH) in
Atlanta. BAX 326 was recently granted orphan-drug designation by the
U.S. Food and Drug Administration (FDA), a special status given to a
product that when approved, will address an unmet need for people with a
rare disease or condition. Data supporting the prophylaxis indication,
which is the basis for the orphan drug designation, was included in the
biologics license application (BLA).

''With only one recombinant protein currently available to treat
hemophilia B, it was important to focus research efforts on finding
another option for patients with this debilitating disease,'' said lead
investigator Jerzy Windyga, MD, PhD, Institute of Hematology and Blood
Transfusion, Warsaw, Poland. ''In this study, more than 40 percent of
patients using BAX 326 as a prophylactic treatment experienced no
bleeds, an important finding given the potentially harmful impact of
bleeding episodes for patients.''

The Phase I/III prospective, controlled, multicenter study investigated
the pharmacokinetics, efficacy and safety of BAX 326 in 73
previously-treated patients with severe or moderately severe hemophilia
B. Results from the study showed that twice-weekly prophylactic
treatment with BAX 326 achieved a median annualized bleed rate of 1.99
with 43 percent of patients experiencing no bleeds. The impact of
prophylaxis with BAX 326 also translated into statistically significant
improvements in physical health-related quality of life (HRQoL), as
measured by improvements in the Bodily Pain and Role-Physical domains in
the Short Form Health Survey (SF-36v2) tool. Statistically significant
changes were not seen in the other physical health-related domains or
the mental health domains.

No inhibitors were detected and no cases of anaphylaxis were reported in
any patients. Three treatment-related adverse events were reported in 2
of the 73 (2.7%) patients, all of which were mild and transient:
dysgeusia (distortion of the sense of taste) occurred twice in one
patient and pain in extremity occurred once in another patient. More
than 70 percent of subjects (56 of 73) had 50 or more exposure days to
BAX 326 during the study.

''As a company committed to advancing care for people with hemophilia
for more than 60 years, Baxter continues to deliver significant
innovation in the field of hemophilia. If it is approved, BAX 326 will
expand treatment options for patients,'' said Prof. Harmut J. Ehrlich,
M.D., vice president of global research and development in Baxter’s
BioScience business.

To continue on its path of innovation, earlier this year Baxter
announced a partnership with Chatham Therapeutics, LLC to develop a gene
therapy-based treatment for hemophilia B. Gene therapy represents
another important area of research that may become the future of
hemophilia B treatment.

About Hemophilia B

Hemophilia B is the second most common type of hemophilia (also known as
Christmas disease) and is the result of insufficient amounts of clotting
factor IX, a naturally occurring protein in blood that controls bleeding.1
Approximately 25,000 people worldwide, including more than 4,000 in the
U.S., have been diagnosed with hemophilia B. 2 Hemophilia B
is often a debilitating, chronic disease with complications that include
bleeding episodes, hemophilic arthropathy (bleeding into a joint) and
hospitalization.3

About Baxter in Hemophilia

Baxter has more than 60 years experience in hemophilia and has
introduced a number of therapeutic firsts for hemophilia patients.
Baxter has the broadest portfolio of hemophilia treatments in the
industry and is able to meet individual therapy choices, providing a
range of options at each treatment stage. The company’s work is focused
on optimizing hemophilia care and improving the lives of people living
with hemophilia A and B worldwide.

About Baxter International Inc.

Baxter International Inc., through its subsidiaries, develops,
manufactures and markets products that save and sustain the lives of
people with hemophilia, immune disorders, cancer, infectious diseases,
kidney disease, trauma and other chronic and acute medical conditions.

This release includes forward-looking statements concerning BAX 326,
an investigational recombinant factor IX (rFIX) protein, as well as
Baxter’s partnership with Chatham Therapeutics, LLC to develop a gene
therapy-based treatment for hemophilia B.The statements are
based on assumptions about many important factors, including the
following, which could cause actual results to differ materially from
those in the forward-looking statements: satisfaction of regulatory and
other requirements; actions of regulatory bodies and other governmental
authorities; additional clinical results; changes in laws and
regulations; product quality or patient safety issues; and other risks
identified in Baxter's most recent filing on Form 10-K and other SEC
filings, all of which are available on Baxter's website. Baxter does not
undertake to update its forward-looking statements.

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